CA2493949C - Petites molecules modifiees d'arn d'interference et leurs procedes d'utilisation - Google Patents
Petites molecules modifiees d'arn d'interference et leurs procedes d'utilisation Download PDFInfo
- Publication number
- CA2493949C CA2493949C CA2493949A CA2493949A CA2493949C CA 2493949 C CA2493949 C CA 2493949C CA 2493949 A CA2493949 A CA 2493949A CA 2493949 A CA2493949 A CA 2493949A CA 2493949 C CA2493949 C CA 2493949C
- Authority
- CA
- Canada
- Prior art keywords
- sirna
- virus
- rna
- hepatitis
- modified
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Expired - Fee Related
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Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1131—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
- A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/20—Antivirals for DNA viruses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/335—Modified T or U
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
- Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
- Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
- Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
- Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change
Priority Applications (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CA 2884658 CA2884658A1 (fr) | 2002-07-26 | 2003-07-25 | Petites molecules modifiees d'arn d'interference et leurs procedes d'utilisation |
Applications Claiming Priority (7)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US39860502P | 2002-07-26 | 2002-07-26 | |
US60/398,605 | 2002-07-26 | ||
US46183803P | 2003-04-11 | 2003-04-11 | |
US60/461,838 | 2003-04-11 | ||
US47023003P | 2003-05-14 | 2003-05-14 | |
US60/470,230 | 2003-05-14 | ||
PCT/US2003/023104 WO2004011647A1 (fr) | 2002-07-26 | 2003-07-25 | Petites molécules modifiées d'arn d'interférence et leurs procédés d'utilisation |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA 2884658 Division CA2884658A1 (fr) | 2002-07-26 | 2003-07-25 | Petites molecules modifiees d'arn d'interference et leurs procedes d'utilisation |
Publications (2)
Publication Number | Publication Date |
---|---|
CA2493949A1 CA2493949A1 (fr) | 2004-02-05 |
CA2493949C true CA2493949C (fr) | 2015-06-02 |
Family
ID=32397947
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA 2884658 Abandoned CA2884658A1 (fr) | 2002-07-26 | 2003-07-25 | Petites molecules modifiees d'arn d'interference et leurs procedes d'utilisation |
CA2493949A Expired - Fee Related CA2493949C (fr) | 2002-07-26 | 2003-07-25 | Petites molecules modifiees d'arn d'interference et leurs procedes d'utilisation |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA 2884658 Abandoned CA2884658A1 (fr) | 2002-07-26 | 2003-07-25 | Petites molecules modifiees d'arn d'interference et leurs procedes d'utilisation |
Country Status (9)
Country | Link |
---|---|
US (4) | US20050058982A1 (fr) |
EP (1) | EP1532248B1 (fr) |
JP (2) | JP4709545B2 (fr) |
AT (1) | ATE427352T1 (fr) |
AU (1) | AU2003261231A1 (fr) |
CA (2) | CA2884658A1 (fr) |
DE (1) | DE60326961D1 (fr) |
ES (1) | ES2322145T3 (fr) |
WO (1) | WO2004011647A1 (fr) |
Families Citing this family (75)
Publication number | Priority date | Publication date | Assignee | Title |
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US8202979B2 (en) * | 2002-02-20 | 2012-06-19 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid |
US20030166282A1 (en) * | 2002-02-01 | 2003-09-04 | David Brown | High potency siRNAS for reducing the expression of target genes |
US20060009409A1 (en) * | 2002-02-01 | 2006-01-12 | Woolf Tod M | Double-stranded oligonucleotides |
CA2474910A1 (fr) | 2002-02-01 | 2003-08-07 | Sequitur, Inc. | Compositions oligonucleotidiques presentant une efficacite amelioree |
US20090192105A1 (en) * | 2002-02-20 | 2009-07-30 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF INTERCELLULAR ADHESION MOLECULE (ICAM) GENE EXPRESSION USING SHORT INTERFERING NUCELIC ACID (siNA) |
JP2005517427A (ja) * | 2002-02-20 | 2005-06-16 | サーナ・セラピューティクス・インコーポレイテッド | 短干渉核酸(siNA)を用いるC型肝炎ウイルス(HCV)遺伝子発現のRNA干渉媒介性阻害 |
US20100075423A1 (en) * | 2002-06-12 | 2010-03-25 | Life Technologies Corporation | Methods and compositions relating to polypeptides with rnase iii domains that mediate rna interference |
US20040248094A1 (en) * | 2002-06-12 | 2004-12-09 | Ford Lance P. | Methods and compositions relating to labeled RNA molecules that reduce gene expression |
EP1532271A4 (fr) * | 2002-06-12 | 2006-10-18 | Ambion Inc | Methodes et compositions relatives a des polypeptides a domaines rnase iii mediant des interferences par l'arn |
ES2322145T3 (es) | 2002-07-26 | 2009-06-17 | Novartis Vaccines And Diagnostics, Inc. | Moleculas pequeñas modificadas de adn inerferente y procedimiento de uso. |
US20040029275A1 (en) * | 2002-08-10 | 2004-02-12 | David Brown | Methods and compositions for reducing target gene expression using cocktails of siRNAs or constructs expressing siRNAs |
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EP1633770B1 (fr) | 2003-06-13 | 2015-04-29 | Alnylam Europe AG | Acide ribonucleique double brin presentant une efficacite accrue dans un organisme |
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WO2005047351A1 (fr) * | 2003-10-28 | 2005-05-26 | Basell Poliolefine Italia S.R.L. | Composants et catalyseurs pour la polymerisation d'olefines |
WO2005087926A2 (fr) | 2004-03-05 | 2005-09-22 | Benitec, Inc. | Cassettes d'expression de promoteurs multiples assurant l'apport simultane d'agents arni |
US20070265220A1 (en) | 2004-03-15 | 2007-11-15 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
AU2005222965B8 (en) | 2004-03-15 | 2010-07-01 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
US20050244869A1 (en) * | 2004-04-05 | 2005-11-03 | Brown-Driver Vickie L | Modulation of transthyretin expression |
WO2005111219A1 (fr) * | 2004-04-16 | 2005-11-24 | University Of Washington | Procedes et vecteurs d'expression d'arnic |
MXPA06012076A (es) * | 2004-04-20 | 2007-01-25 | Nastech Pharm Co | Metodos y composiciones para mejorar el suministro de arn bicatenario o un acido nucleico hibrido bicatenario para regular la expresion genetica en celulas de mamifero. |
CA2568013C (fr) | 2004-05-27 | 2015-11-24 | Alnylam Pharmaceuticals, Inc. | Acide ribonucleique double brin resistant aux nucleases |
US8361976B2 (en) | 2004-07-09 | 2013-01-29 | University Of Massachusetts | Therapeutic alteration of transplantable tissues through in situ or ex vivo exposure to RNA interference molecules |
US7297786B2 (en) | 2004-07-09 | 2007-11-20 | University Of Iowa Research Foundation | RNA interference in respiratory epitheial cells |
US7582744B2 (en) * | 2004-08-10 | 2009-09-01 | Alnylam Pharmaceuticals, Inc. | Chemically modified oligonucleotides |
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CA2581651C (fr) * | 2004-10-01 | 2014-12-16 | Novartis Vaccines And Diagnostics, Inc. | Arn modifie marque avec du cholesterol |
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JP6893785B2 (ja) | 2013-10-22 | 2021-06-23 | トランスレイト バイオ, インコーポレイテッド | mRNAのCNS送達及びその使用方法 |
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US9943595B2 (en) | 2014-12-05 | 2018-04-17 | Translate Bio, Inc. | Messenger RNA therapy for treatment of articular disease |
AU2016233135B2 (en) | 2015-03-19 | 2021-07-08 | Translate Bio, Inc. | mRNA therapy for pompe disease |
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-
2003
- 2003-07-25 ES ES03771767T patent/ES2322145T3/es not_active Expired - Lifetime
- 2003-07-25 AT AT03771767T patent/ATE427352T1/de not_active IP Right Cessation
- 2003-07-25 CA CA 2884658 patent/CA2884658A1/fr not_active Abandoned
- 2003-07-25 WO PCT/US2003/023104 patent/WO2004011647A1/fr active Application Filing
- 2003-07-25 DE DE60326961T patent/DE60326961D1/de not_active Expired - Lifetime
- 2003-07-25 CA CA2493949A patent/CA2493949C/fr not_active Expired - Fee Related
- 2003-07-25 AU AU2003261231A patent/AU2003261231A1/en not_active Abandoned
- 2003-07-25 JP JP2004524748A patent/JP4709545B2/ja not_active Expired - Fee Related
- 2003-07-25 US US10/626,879 patent/US20050058982A1/en not_active Abandoned
- 2003-07-25 EP EP20030771767 patent/EP1532248B1/fr not_active Expired - Lifetime
-
2006
- 2006-06-15 JP JP2006165652A patent/JP4718379B2/ja not_active Expired - Fee Related
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2011
- 2011-03-30 US US13/075,944 patent/US20120122962A1/en not_active Abandoned
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2013
- 2013-03-12 US US13/796,606 patent/US9139833B2/en not_active Expired - Fee Related
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WO2004011647A8 (fr) | 2004-06-10 |
ATE427352T1 (de) | 2009-04-15 |
EP1532248B1 (fr) | 2009-04-01 |
US20050058982A1 (en) | 2005-03-17 |
EP1532248A1 (fr) | 2005-05-25 |
CA2884658A1 (fr) | 2004-02-05 |
JP2005533517A (ja) | 2005-11-10 |
AU2003261231A1 (en) | 2004-02-16 |
US9139833B2 (en) | 2015-09-22 |
ES2322145T3 (es) | 2009-06-17 |
WO2004011647A1 (fr) | 2004-02-05 |
US20150361432A1 (en) | 2015-12-17 |
JP4718379B2 (ja) | 2011-07-06 |
JP4709545B2 (ja) | 2011-06-22 |
CA2493949A1 (fr) | 2004-02-05 |
AU2003261231A8 (en) | 2004-02-16 |
DE60326961D1 (de) | 2009-05-14 |
JP2006320327A (ja) | 2006-11-30 |
US20140073683A1 (en) | 2014-03-13 |
US20120122962A1 (en) | 2012-05-17 |
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